Current Progress in Therapeutic Gene Editing for Monogenic Diseases [PDF]
, 2016 Programmable nucleases allow defined alterations in the genome with ease-of-use, efficiency, and specificity. Their availability has led to accurate and widespread genome engineering, with multiple applications in basic research, biotechnology, and ...
Moore, Marc +2 more
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May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells
Cells, 2017 In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and ...
Nicholas Brookhouser +3 more
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Strategies for mitochondrial gene editing
Computational and Structural Biotechnology Journal, 2021 Mitochondria, as the energy factory of cells, participate in metabolism processes and play a critical role in the maintenance of human life activities. Mitochondria belong to semi-automatic organelles, which have their own genome different from nuclear ...
Xingbo Yang +4 more
doaj
Development of Medicinal Products Based on Gene-Editing Technology: Regulatory Practices
Регуляторные исследования и экспертиза лекарственных средств, 2023 Somatic cell genome-editing systems are the most recent gene therapy technology to treat patients with monogenic hereditary cancer or HIV. Gene editing allows for changing or completely removing a defective gene with regularly interspaced short ...
N. S. Pokrovsky +3 more
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Current Status of Production of Transgenic Livestock by Genome Editing Technology
Journal of Animal Reproduction and Biotechnology, 2019 The Transgenic livestock can be useful for the production of disease-resistant animals, pigs for xenotranplantation, animal bioreactor for therapeutic recombinant proteins and disease model animals.
Da Som Park +3 more
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Magnetic separation and antibiotics selection enable enrichment of cells with ZFN/TALEN-induced mutations. [PDF]
PLoS ONE, 2013 The ability to enrich cells with targeted mutations greatly facilitates the process of using engineered nucleases, including zinc-finger nucleases and transcription activator-like effector nucleases, to construct such cells.
Hyojin Kim +5 more
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Bacteria herald a new era of gene editing. [PDF]
, 2013 The demonstration that nucleases guided by bacterial RNA can disrupt human genes represents a landmark in the rapidly developing field of genome ...
Segal, David J
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Robust method for TALEN-edited correction of pF508del in patient-specific induced pluripotent stem cells [PDF]
, 2016 Immunofluorescence detection of stem cell markers in CFhIPS cell lines. Immunostaining for stem cell markers in ten of the 49 lines originally generated. Markers are indicated in the panel and are distributed in columns.
María Vicenta Camarasa +1 more
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Efficient Gene Transfer and Gene Editing in Sterlet (Acipenser ruthenus)
Frontiers in Genetics, 2018 The sturgeon (Acipenseriformes) is an important farmed species because of its economical value. However, neither gene transfer nor gene editing techniques have been established in sturgeon for molecular breeding and gene functional study until now.
Ji Chen +9 more
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Integrate CRISPR/Cas9 for protein expression of HLA-B*38:68Q via precise gene editing. [PDF]
, 2019 The determination of null- or low-expressed HLA alleles is clinically relevant in both hematopoietic stem cell transplantation and solid organ transplantation.
Reed, Elaine F +2 more
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